Recently, the U.S. Food and Drug Administration (FDA) granted Priority Review to relugolix for the treatment of advanced prostate cancer; Fast Track designation to a radioimmunotherapeutic treatment for marginal zone lymphoma; and Orphan Drug designation to a novel monoclonal antibody for pancreatic cancer.
Priority Review for Relugolix in Advanced Prostate Cancer
The FDA accepted a new drug application for Priority Review for once-daily oral relugolix (120 mg) for the treatment of men with advanced prostate cancer. The FDA has set a target action date of December 20, 2020, under the Prescription Drug User Fee Act (PDUFA).
If approved, relugolix would be the first and only oral gonadotropin-releasing hormone receptor antagonist treatment for men with advanced prostate cancer.
The randomized, open-label, parallel-group, multinational phase III HERO trial was designed to evaluate the safety and efficacy of relugolix in over 900 men with androgen-sensitive advanced prostate cancer who required at least 1 year of continuous androgen-deprivation therapy. Men were randomly assigned 2:1 to receive a single loading dose of relugolix at 360 mg followed by relugolix at 120 mg once daily, or to treatment with leuprolide acetate 3-month depot injection, respectively.
Relugolix met the primary efficacy endpoint, with 96.7% of men treated with relugolix achieving sustained testosterone suppression to castrate levels (< 50 ng/dL) through 48 weeks vs 88.8% of men treated with leuprolide acetate. Relugolix also met all six key secondary endpoints, demonstrating superiority to leuprolide acetate in rapid and profound suppression of testosterone and prostate-specific antigen response, in addition to improved testosterone recovery after discontinuation of treatment. Men in the relugolix group had a 54% lower risk of major adverse cardiovascular events compared to men in the leuprolide acetate group (2.9% vs 6.2%). In men with a reported history of major adverse cardiovascular events, the relugolix group had 80% fewer events reported compared to the leuprolide acetate group (3.6% vs 17.8%). The overall incidence of adverse events in the relugolix and leuprolide acetate groups was comparable (92.9% vs 93.5%).
Data from an additional key secondary endpoint, castration resistance–free survival, are expected later in 2020.
Lu-177 Lilotomab Satetraxetan Receives Fast Track Designation for Marginal Zone Lymphoma
The FDA granted Fast Track designation to Lu-177 lilotomab satetraxetan for the treatment of adult patients with relapsed or refractory marginal zone lymphoma who have received at least two prior systemic therapies.
The agent has demonstrated activity in nine patients with marginal zone lymphoma in the phase I/IIa LYMRIT 37-01 trial. Treatment with Lu-177 lilotomab satetraxetan resulted in a 78% overall response rate and 44% complete response in the nine patients. This followed a single administration of the agent in this heavily pretreated group of patients with advanced disease.
Lu-177 lilotomab satetraxetan has received Orphan Drug designation in the European Union for the treatment of marginal zone lymphoma; its manufacturer has applied for the equivalent designation in the United States.
Orphan Drug Designation for PBP1510 in Pancreatic Cancer
The FDA granted Orphan Drug Designation to a first-in-class anti-PAUF monoclonal antibody, PBP1510, for the treatment of pancreatic cancer.
Limited efficacy of treatment modalities and rapid progression of pancreatic cancer can be partly explained by the overexpression of a gene called pancreatic adenocarcinoma upregulated factor (PAUF) found in majority of pancreatic cancers. PAUF plays an important role in disease progression, but no targeted molecular therapy against PAUF currently exists. PBP1510 is envisioned to provide significant benefit for patients with PAUF-positive pancreatic cancer.
The manufacturer has also filed for Orphan Drug designation status in the European Union.
